Huntington’s disease is an obvious target for gene therapies. Caused by long repeats in a single specific gene and the…
Technology
This is like giving people a molecular coat of armor. Fyodor Urnov Two teams of scientists in the USA work…
We envision Cas13 as a research tool to explore many aspects of viral biology in human cells. It could also…
A year ago, we covered preliminary results of ultra-personalized gene therapy for a rare form of Batten disease. Team behind the treatment…
These are sequences that we don’t interrogate today because they are not in the human reference genome—so if they are…
The two eyes communicate not only in disease, but also in response to treatment. David J. Calkins A study on…
Contrary to popular notion, evolution is more of a pianist than a (blind) watchmaker: fiddles mostly with gene regulation, trying…
A new approach, utilizing lipid vesicles, successfully targeted genes of human papilloma virus (HPV) and completely cured mice from cervical…
A multi-year study of genetically modified cattle proved that its offspring is physically healthy, and preserves introduced trait – lack…
Mendelian disorders – although genetically simple – can pose significant challenges in the development of gene therapy. In the case…